Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 6:32 PM
Ignite Modification Date:
2025-12-24 @ 6:32 PM
NCT ID:
NCT06137157
Brief Summary:
The objectives of this clinical trial are to evaluate the safety and tolerability of topically applied ATR12-351, to understand what the body does to ATR12-351, and to observe treatment benefits of the drug in approximately 12 adult patients with Netherton Syndrome (NS). ATR12-351 will be applied to skin lesions on one side of the body, while the vehicle control will be applied to similar lesion on the other side of the body twice daily for 2 weeks.
Detailed Description:
Netherton syndrome (NS) is a rare but severe autosomal recessive disease that affects the skin, hair, and immune system. NS presents at birth with red skin covered by fine scales, a specific hair shaft abnormality ("bamboo hair"), atopic lesions, and elevated systemic immunoglobulin E (IgE) levels. Patients often develop allergies and asthma, and the unfortunate prognosis for infants with NS is poor, with 10% mortality in the first 6 months of life and recurrent infections. There are no FDA-approved treatment options for NS. NS is caused by mutations in in the SPINK5 gene encoding the serine protease inhibitor lymphoepithelial Kazal-type related inhibitor (LEKTI). Absence of LEKTI causes unregulated activity of proteases in the kallikrein subfamily, causing excessive desquamation of skin.
ATR12-351 is a topical ointment containing a lyophilized version of a live biotherapeutic product (LBP), Staphylococcus epidermidis (SE) strain designated SE351. This strain has been modified to be auxotrophic and to express recombinant human LEKTI protein (rhLEKTI). ATR12-351 is intended to address the underlying cause of NS by replacing deficient/dysfunctional LEKTI. The treatment consists of applying ATR12-351 to affected areas, where rhLEKTI produced by ATR12-351 will counter the dysregulated skin serine protease activity observed in NS patients.
This first-in-human study is a randomized, double-blind, vehicle-controlled clinical study to assess the safety, tolerability, and pharmacokinetics (PK) of topical ATR12-351 in approximately 12 adult Netherton syndrome patients who will serve as their own control. This study will include a 14-day treatment period to investigate the safety and tolerability of ATR12-351 applied to the skin and will be followed a total of 84 days for safety. The results of this first-in-human clinical study will establish safety and tolerability as well as initial efficacy of ATR12-351 application in Netherton syndrome patients.
Study:
NCT06137157
Study Brief:
Protocol Section:
NCT06137157